Clinical Trials for Melanoma: Your Input Needed

I am a Stage IV melaonoma survivor and melanoma activist. I have been invited to participate in the Patient-Centered Clinical Trial Summit at the Partnership in Clinical Trials Conference. This gives me a unique opportunity to present a patient perspective to key stakeholders in Pharma, the CRO industry and the regulatory community. I am looking for additional patient input concerning the design, conduct, financing and analysis of clinical trials as well as the regulatory process.

I am a Stage IV melaonoma survivor and melanoma activist. I have been invited to participate in the Patient-Centered Clinical Trial Summit at the Partnership in Clinical Trials Conference. This gives me a unique opportunity to present a patient perspective to key stakeholders in Pharma, the CRO industry and the regulatory community. I am looking for additional patient input concerning the design, conduct, financing and analysis of clinical trials as well as the regulatory process.

The entire Translational Research Model in the US and internationally is under scrutiny by groups at home and abroad. ( see for example: the Conference on Clinical Care Research sponsored by the Friends of Cancer Research in November of 2012). We are close to significant changes in how our FDA will regulate Breakthrough Therapies under PDFUA 5. However, the international picture is not as good: requirements for approval such as  endpoint selection, randomization, comparator arms, use of placebos, and etc. are all based on antiquated notions of what constiutes scientific proof. They treat all diseases alike, they were designed to evaluate synthetic chemical treatments. They do not recognize that critical diseases like metastatic melanoma require a different approach to clinical trials because too many people are dying and because new targeted and immunologic therapies may not yet cure but have higher response rates, more durable responses, more tolerability and more practical benefits than the Starndard of Care. With the globalization of the pharmeceutical marketplace and the globilization of clinical trials to reduce costs and problems of patient recruitment, the problem of  differential standards has been exacerbated. I intend to use as an example the recently announced BMS Phase 3 trial NCT01721772) of anti-PD-1 (BMS-036558). It is the breakthrough therapy that saved my life (Extended 1B Trial) and an excellent example of the need to change regulatory practices and trial design now. As I am fond of saying , we need anti-PD-1 and we need it now!  If we want participatory medicine, we must participate not only at the level of personal choice but at the policy level.

As I prepare my thoughts, I need to know yours. You may respond here or to me directly at [email protected] if you desire your comments to be kept private.

Alan